Mueller, Christian

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α 1 -Antitrypsin: Interim Results

Flotte, T. R., Trapnell, B. C., Humphries, M., Carey, B., Calcedo, R., Rouhani, F., Campbell-Thompson, M., Yachnis, A. T., Sandhaus, R. A., McElvaney, N. G., et al. (2011) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 335

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Brantly, M. L., Chulay, J. D., Wang, L., Mueller, C., Humphries, M., Spencer, L. T., Rouhani, F., Conlon, T. J., Calcedo, R., Betts, M. R., et al. (2009) · Proceedings of the National Academy of Sciences

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 327

Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

Zhang, H., Yang, B., Mu, X., Ahmed, S. S., Su, Q., He, R., Wang, H., Mueller, C., Sena-Esteves, M., Brown, R., et al. (2011) · Molecular Therapy

Virus-based gene therapy researchBarrier Structure and Function Studies

Cited by 291

SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS

Mueller, C., Berry, J. D., McKenna-Yasek, D. M., Gernoux, G., Owegi, M. A., Pothier, L. M., Douthwright, C. L., Gelevski, D., Luppino, S. D., Blackwood, M., et al. (2020) · New England Journal of Medicine

Amyotrophic Lateral Sclerosis ResearchNeurogenetic and Muscular Disorders Research

Cited by 264

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

Alterman, J. F., Godinho, B. M. D. C., Hassler, M. R., Ferguson, C. M., Echeverria, D., Sapp, E., Haraszti, R. A., Coles, A. H., Conroy, F., Miller, R., et al. (2019) · Nature Biotechnology

RNA Interference and Gene DeliveryDNA and Nucleic Acid Chemistry

Cited by 227

Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression

Mueller, C., Chulay, J. D., Trapnell, B. C., Humphries, M., Carey, B., Sandhaus, R. A., McElvaney, N. G., Messina, L., Tang, Q., Rouhani, F. N., et al. (2013) · Journal of Clinical Investigation

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 169

AAV gene therapy for Tay-Sachs disease

Flotte, T. R., Cataltepe, O., Puri, A., Batista, A. R., Moser, R., McKenna-Yasek, D., Douthwright, C., Gernoux, G., Blackwood, M., Mueller, C., et al. (2022) · Nature Medicine

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 143

5 Year expression and neutrophil defect repair after gene therapy in alpha-1 antitrypsin deficiency

Mueller, C., Gernoux, G., Gruntman, A. M., Borel, F., Reeves, E. P., Calcedo, R., Rouhani, F. N., Yachnis, A., Humphries, M., Campbell-Thompson, M., et al. (2017) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 112

Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease

Pfister, E. L., DiNardo, N., Mondo, E., Borel, F., Conroy, F., Fraser, C., Gernoux, G., Han, X., Hu, D., Johnson, E., et al. (2018) · Human Gene Therapy

Genetic Neurodegenerative DiseasesRNA Interference and Gene Delivery

Cited by 95

Central Role of IL-23 and IL-17 Producing Eosinophils as Immunomodulatory Effector Cells in Acute Pulmonary Aspergillosis and Allergic Asthma

Guerra, E. S., Lee, C. K., Specht, C. A., Yadav, B., Huang, H., Akalin, A., Huh, J. R., Mueller, C., & Levitz, S. M. (2017) · PLOS Pathogens

Asthma and respiratory diseasesIL-33, ST2, and ILC Pathways

Cited by 88

Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency

Calcedo, R., Somanathan, S., Qin, Q., Betts, M. R., Rech, A. J., Vonderheide, R. H., Mueller, C., Flotte, T. R., & Wilson, J. M. (2017) · Proceedings of the National Academy of Sciences

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 79

Genetic Deficiency of Flavin-Containing Monooxygenase 3 ( Fmo3 ) Protects Against Thrombosis but Has Only a Minor Effect on Plasma Lipid Levels—Brief Report

Shih, D. M., Zhu, W., Schugar, R. C., Meng, Y., Jia, X., Miikeda, A., Wang, Z., Zieger, M., Lee, R., Graham, M., et al. (2019) · Arteriosclerosis, Thrombosis, and Vascular Biology

Pharmacogenetics and Drug MetabolismBlood Coagulation and Thrombosis Mechanisms

Cited by 67

Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity

Xiao, Y., Muhuri, M., Li, S., Qin, W., Xu, G., Luo, L., Li, J., Letizia, A. J., Wang, S. K., Chan, Y. K., et al. (2019) · JCI Insight

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 53

The Promise of Gene Therapy For The Treatment of α-1 Antitrypsin Deficiency

Cruz, P. E., Mueller, C., & Flotte, T. R. (2007) · Pharmacogenomics

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 10